Lexeo Therapeutics Granted FDA Fast Track Designation for LX2006, an AAV-Based Gene Therapy Candidate for the Treatment of Friedreich’s Ataxia Cardiomyopathy
16 April 2024 - 9:30PM
Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic
medicine company dedicated to pioneering treatments for genetically
defined cardiovascular diseases and APOE4-associated Alzheimer’s
disease, today announced the U.S. Food and Drug Administration
(FDA) has granted Fast Track designation to LX2006, the company’s
AAVrh.10hFXN-based gene therapy candidate for the treatment of
Friedreich’s ataxia (FA) cardiomyopathy. LX2006 is designed to
deliver a functional frataxin gene to promote frataxin protein
expression and restore mitochondrial function in myocardial cells.
Fast Track is a process designed to facilitate the development
and expedite the review of new drugs intended to treat serious
conditions and address unmet medical need. This designation was
granted based on available preclinical data. SUNRISE-FA, a Phase
1/2 multicenter, 52-week, dose-ascending, open-label clinical
trial, is ongoing to evaluate the safety and tolerability, as well
as preliminary efficacy, of LX2006 in patients with FA
cardiomyopathy.
“FA cardiomyopathy is the leading cause of death among FA
patients, and there are currently no approved treatment options.
The FDA’s Fast Track designation for LX2006 underscores the
significant unmet need for effective treatment options to address
the cardiac impact of this debilitating disease,” said R. Nolan
Townsend, Chief Executive Officer of Lexeo Therapeutics. “We
believe today’s Fast Track designation, along with the previously
announced Rare Pediatric Disease and Orphan Drug designations
granted to LX2006, will allow for enhanced regulatory interactions
and the potential for this life-improving therapy to reach FA
patients more quickly.”
LX2006 is administered as a one-time intravenous infusion to
patients in at least two ascending-dose cohorts with the potential
for a third cohort. Long-term safety and efficacy will be evaluated
for an additional four years following completion of the initial
year of the trial, resulting in data from a total of five years
post-LX2006 treatment.
About LX2006LX2006 is an AAV-based gene therapy
candidate delivered intravenously for the treatment of FA
cardiomyopathy, the most common cause of mortality in patients with
FA affecting approximately 5,000 patients in the United States.
LX2006 is designed to target the cardiac manifestations of FA by
delivering a functional frataxin gene to promote the expression of
the frataxin protein and restore mitochondrial function in
myocardial cells. In preclinical studies, LX2006 reversed the
cardiac abnormalities in FA disease models and showed improvement
in cardiac function and survival while demonstrating a favorable
safety profile. The FDA has granted Fast Track designation, Rare
Pediatric Disease designation and Orphan Drug designation to LX2006
for the treatment of FA cardiomyopathy.
About Lexeo Therapeutics Lexeo
Therapeutics is a New York City-based, clinical stage genetic
medicine company dedicated to transforming healthcare by applying
pioneering science to fundamentally change how genetically defined
cardiovascular diseases and APOE4-associated Alzheimer’s disease
are treated. Using a stepwise development approach, Lexeo is
leveraging early proof-of-concept functional and biomarker data to
advance a pipeline of cardiovascular and APOE4-associated
Alzheimer’s disease programs.
Cautionary Note Regarding Forward-Looking
StatementsCertain statements in this press release may
constitute “forward-looking statements” within the meaning of the
federal securities laws, including, but not limited to, our
expectations and plans regarding our current product candidates and
programs, including statements regarding the anticipated timing of
the initiation of and results from our clinical trials and other
information that is not historical information. Words such as
“may,” “might,” “will,” “objective,” “intend,” “should,” “could,”
“can,” “would,” “expect,” “believe,” “design,” “estimate,”
“predict,” “potential,” “develop,” “plan” or the negative of these
terms, and similar expressions, or statements regarding intent,
belief, or current expectations, are forward-looking statements.
While Lexeo believes these forward-looking statements are
reasonable, undue reliance should not be placed on any such
forward-looking statements. These forward-looking statements are
based upon current information available to the company as well as
certain estimates and assumptions and are subject to various risks
and uncertainties (including, without limitation, those set forth
in Lexeo’s filings with the U.S. Securities and Exchange Commission
(SEC)), many of which are beyond the company’s control and subject
to change. Actual results could be materially different from those
indicated by such forward looking statements as a result of many
factors, including but not limited to: risks and uncertainties
related to global macroeconomic conditions and related volatility;
expectations regarding the initiation, progress, and expected
results of Lexeo’s preclinical studies, clinical trials and
research and development programs; the unpredictable relationship
between preclinical study results and clinical study results;
delays in submission of regulatory filings or failure to receive
regulatory approval; liquidity and capital resources; and other
risks and uncertainties identified in Lexeo’s Annual Report on Form
10-K for the annual period ended December 31, 2023, filed with the
SEC on March 11, 2024, and subsequent future filings Lexeo may make
with the SEC. New risks and uncertainties may emerge from time to
time, and it is not possible to predict all risks and
uncertainties. Lexeo claims the protection of the Safe Harbor
contained in the Private Securities Litigation Reform Act of 1995
for forward-looking statements. Lexeo expressly disclaims any
obligation to update or alter any statements whether as a result of
new information, future events or otherwise, except as required by
law.
Media Response:Janine Bogris (201) 245-6838
janine.bogris@inizioevoke.com
Investor Response:Stephen Jasper(858)
525-2047stephen@gilmartinir.com
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